Ipsen signs $1.8bn RNA drug alliance with Skyhawk
WALTHAM, Mass., Feb. 28, 2024 /PRNewswire/ -- Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule RNA splicing modifiers, today announced that the Phase 1 multiple ascending dose (MAD) portion of its Phase 1 clinical study has now begun in Australia for the company's SKY-0515 small molecule candidate targeting Huntington's disease (HD). Huntington's is caused by a toxic 'CAG' repeat in the huntingtin (HTT) gene, causing mutated huntingtin protein. SKY-0515 is designed to modify the RNA expression of HTT, reducing the production of mutated protein that leads to disease progression in Huntington's patients.
Once considered impossible, Big Pharma has warmed up to the idea of drugging RNA over the last few years, inking platform deals with companies like Arrakis and Skyhawk. Now, Sanofi and Novartis are reaching a little deeper into their wallets for one of the earliest players in this arena.
A few years after becoming one of the first major biotechs out to drug RNA with small molecules, serial entrepreneur and filmmaker Bill Haney is reeling in another $133 million for Skyhawk Therapeutics. And it sounds like he’s got the S-1 papers in his back pocket.
Aragen Life Sciences (formerly GVK BIO) announced that it has been selected by Skyhawk Therapeutics, a leader in the development of small molecule therapeutics that correct RNA expression, as its partner in India. Through this collaboration, Aragen will provide Skyhawk with various discovery chemistry and biology service solutions.